Funded Projects

Project # Project Title Research Focus Area Research Program Administering IC(s) Institution(s) Investigator(s) Location(s) Year Awarded
Optimization of a Gene Therapy for Chronic Pain in Human DRGs Cross-Cutting Research Small Business Programs NINDS NAVEGA THERAPEUTICS, INC. MORENO, ANA MARIA La Jolla, CA 2021
FOA Title: HEAL INITIATIVE: Development of Therapies and Technologies Directed at Enhanced Pain Management (R43/R44 - Clinical Trial Not Allowed)
FOA Number: NS-20-011

To avoid the reliance on opioids for treatment of pain, researchers are investigating alternative approaches to disrupt the transmission of pain signals by specialized neurons in the body, such as dorsal root ganglion neurons in the spinal cord. Molecules called voltage-gated sodium channels that are located in the membranes of dorsal root ganglion neurons are essential for transmission of the pain signals. People carrying a specific variant of these channels, NaV1.7, are insensitive to pain; therefore, strategies to block this particular channel might help in the development of non-addictive pain treatment approaches. Navega Therapeutics is developing an innovative gene therapy that specifically targets NaV1.7. Using studies in human cell lines, they will identify the best designs to then test this gene therapy approach in human dorsal root ganglion neurons.

Repression of Sodium Channels via a Gene Therapy for Treatment of Chronic Neuropathic Pain Cross-Cutting Research Small Business Programs NINDS NAVEGA THERAPEUTICS, INC. MORENO, ANA MARIA; ALEMAN GUILLEN, FERNANDO San Diego, CA 2019
FOA Title: PHS 2018-02 Omnibus Solicitation of the NIH, CDC, and FDA for Small Business Innovation Research Grant Applications (Parent SBIR [R43/R44] Clinical Trial Not Allowed)
FOA Number: PA-18-574

Voltage-gated sodium channels are responsible for the transmission of pain signals. Nine genes have been identified, each having unique properties and tissue distribution patterns. Genetic studies have correlated a hereditary loss-of-function mutation in one human Na+ channel isoform – ?Na?V?1.7 – with a rare genetic disorder known as Congenital Insensitivity to Pain (CIP). Individuals with CIP are not able to feel pain without any significant secondary alteration. Thus, selective inhibition of ?Na?V?1.7 in normal humans could recapitulate the phenotype of CIP. This research team developed a non-permanent gene therapy to target pain that is non-addictive (because it targets a non-opioid pathway), highly specific (only targeting the gene of interest), and long-term lasting (around 3 weeks in preliminary assays in mice). During this Phase I , the team will 1) test additional pain targets ?in vitro?, and 2) evaluate the new targets ?in vivo ?in mice models of inflammatory and neuropathic pain.